EU regulators have moved to widen use of two medicines in areas with clear unmet need, backing one label expansion for adolescent iron deficiency and another for hemophilia prophylaxis regardless of inhibitor status.

At its March 2026 meeting, the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) issued positive opinions recommending updates to the marketing authorizations for Feraccru (ferric maltol, Norgine B.V.) and Hympavzi (marstacimab, Pfizer Europe MA EEIG).

If approved by the European Commission, the changes would expand treatment options for iron deficiency in adolescents and broaden routine prophylaxis for hemophilia A and B in patients with or without inhibitors.

Pediatric Expansion for Iron Deficiency 

The first update for Feraccru extends its indication to adolescents aged 12 years or older with iron deficiency. This variation would amend the existing indication for adult use only.

Iron deficiency is a common condition characterized by low levels of iron in the body, which can lead to anemia, fatigue, and impaired cognitive or physical function. Diagnosis should be confirmed with lab tests, with other causes of anemia excluded. 

Feraccru contains ferric maltol, which enhances iron absorption while limiting gastrointestinal side effects. It is taken orally as a capsule, twice daily, preferably on an empty stomach. The duration of treatment varies with the severity of iron deficiency, but therapy is typically continued for at least 12 weeks.

The recommendation is supported by clinical evidence from phase 1 and phase 3 studies. 

A phase 1 pharmacokinetic study in children aged 10-17 demonstrated rapid absorption of ferric maltol with peak maltol glucuronide by 1-3 hours, dose‑dependent increases in serum iron and transferrin saturation, modest hemoglobin rises of about 0.3-0.7 g/dL over 10 days, and good tolerability across doses.

A phase 3 comparative trial in children and adolescents showed clinically meaningful hemoglobin increases by about 1.1 g/dL at week 12 with ferric maltol oral suspension, along with a favorable gastrointestinal tolerability profile relative to ferrous sulfate, informing both safety and efficacy in the target age group. A crossover pharmacokinetic study in healthy adults established bioequivalence of oral suspension and capsules under fed conditions, supporting flexible formulation use in clinical practice.

The most common side effects of Feraccru are gastrointestinal symptoms such as abdominal pain, flatulence, constipation, abdominal discomfort and distension, and diarrhea.

Feraccru is contraindicated in iron overload disorders such as hemochromatosis or in patients receiving repeated blood transfusions. 

Broadened Prophylaxis Across Inhibitor Status 

The second recommendation broadens the use of Hympavzi for routine prophylaxis of bleeding episodes in patients aged 12 years or older weighing at least 35 kg with hemophilia A or hemophilia B, with or without factor VIII or IX inhibitors. Previously, its authorized EU use was limited to patients without inhibitors and with severe disease.

About 20% of hemophilia A and 3% of hemophilia B patients develop inhibitors, increasing bleeding risk, treatment burden, and costs. Unlike factor VIII or IX replacement treatments, Hympavzi acts by targeting tissue factor pathway inhibitor (TFPI), a natural inhibitor of clot formation. By binding the Kunitz 2 domain of TFPI, it may restore hemostatic balance.

The CHMP’s decision was based on efficacy and safety data from the inhibitor cohort of the phase 3 BASIS trial. The study evaluated marstacimab in participants aged 12 to under 75 years with severe hemophilia A or moderately severe to severe hemophilia B, with or without inhibitors.

Once-weekly subcutaneous marstacimab reduced treated annualized bleeding rates by about 93% in 48 patients with hemophilia A or B with inhibitors, with a mean annualized bleeding rate of 1.39 compared with 19.78 on prior on-demand therapy. 

Hympavzi commonly causes injection‑site reactions, headache, high blood pressure, and itching. Rare but serious adverse events include severe rash and thromboembolic events.

For both newly recommended extensions, detailed recommendations for use will be provided in the updated Summary of Product Characteristics, to be published on the EMA website in all official European Union languages after the European Commission issues a decision.